Tofersen: First Gene-Targeted ALS Therapy Brings Hope for SOD1 Patients

Tofersen (Qalsody®), approved by FDA in 2023, is the first gene-targeted ALS therapy. This antisense oligonucleotide targets SOD1 mRNA to reduce toxic SOD1 protein. Despite not meeting primary endpoint in VALOR trial, 55% reduction in serum NfL was accepted as strong evidence of neuroprotection. The ATLAS trial for presymptomatic SOD1 carriers is ongoing.